Overview

Phase III Randomized Study of Sodium Dichloroacetate in Children With Congenital Lactic Acidosis

Status:
Completed
Trial end date:
2002-09-01
Target enrollment:
0
Participant gender:
All
Summary
OBJECTIVES: I. Compare the safety of sodium dichloroacetate (DCA) vs placebo in children with congenital lactic acidosis. II. Determine the quality of life of these patients. III. Determine the pharmacokinetics and metabolic fate of DCA over the course of drug administration in these patients.
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
University of Florida
Criteria
PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Diagnosis of congenital lactic acidosis (CLA) meeting the following criteria: Three basal
venous lactates at least 2.5 mM, arterial lactates at least 2.0 mM, or CSF lactates at
least 2.5 mM OR any combination of these, obtained over at least 1 month and within 6
months OR Increase in blood lactate at least 1.0 mM over basal following a carbohydrate
meal challenge

AND

Enzymatic or molecular genetic proof of a defect of pyruvate dehydrogenase complex, one or
more respiratory chain enzymes, or a Krebs cycle enzyme OR Over production of C14-lactate
from C14-glucose by cultured skin fibroblasts

AND

Ability to withstand an 8 hour (if 2 years and under) or 12 hour (if over 2 years) fast
without developing hypoglycemia (blood glucose less than 50 mg/dL)

No secondary lactic acidosis due to impaired oxygenation or circulation

No hyperlactatemia associated with proven biotinidase deficiency (biotin responsive CLA) or
with enzyme deficiencies of gluconeogenesis

No primary, defined organic acidurias other than lactic acidosis, for which effective
therapy is available (e.g., propionic aciduria)

No primary disorders of amino acid metabolism or fatty acid oxidation

No malabsorption syndromes associated with D-lactic acidosis

--Prior/Concurrent Therapy--

No chronic dialysis

--Patient Characteristics--

Hepatic: No primary hepatic disease unrelated to CLA

Renal: Creatinine less than 1.2 mg/dL OR Creatinine clearance at least 60 mL/min

Other: No concurrent infection or fever